1. Volume 25, Issue 5, Pages 1155-1162 (May 2017)
Hematopoietic Stem Cell Gene Therapy for Storage Disease: Current and New Indications 
Alessandra Biffi 
Molecular Therapy 
Volume 25, Issue 5, Pages (May 2017)
DOI: /j.ymthe
Copyright © 2017 The American Society of Gene and Cell Therapy Terms and Conditions

2. Figure 1 Lysosomal Enzyme Deficiency and Its Consequences
The defective activity of a lysosomal enzyme (E) determines the accumulation of undegraded substrates in LSD cells, leading to the activation of a pathologic cascade of events.
Molecular Therapy  , DOI: ( /j.ymthe )
Copyright © 2017 The American Society of Gene and Cell Therapy Terms and Conditions

3. Figure 2 The Mechanisms of Cross-Correction
Functional enzyme secreted by wild-type cells is taken up and sorted to the lysosomal compartment of the LSD cells through the mannose-6-phosphate receptor (M6P-R) pathway. This leads to restoration of substrate metabolism.
Molecular Therapy  , DOI: ( /j.ymthe )
Copyright © 2017 The American Society of Gene and Cell Therapy Terms and Conditions